Our genes are large and there can be many different mutations within a single gene. Each mutation can cause a very different outcome in terms of cause of disease, disease symptoms and severity. At n-Lorem, we not only target the specific gene, but design our program to address the specific gene mutation for each patient.
It is our hope that, as more genome sequencing is conducted, more patients will be identified that have a gene mutation that fits a program we are already working on. For those patients, we will already be well ahead in discovering and developing a potential experimental ASO medicine.